Evaluate the Therapeutic Effects and Safety of ALK4290 in Patients With Refractory Wet Age-Related Macular Degeneration

Phase 2

Completed

Conditions: Wet Age-related Macular Degeneration

Interventions: Drug: ALK4290

Registration Number: NCT03558074

Lead Sponsor: Alkahest, Inc.

Brief Summary: This study will evaluate the therapeutic effects and safety of 800 mg ALK4290 administered daily over a 6-week dosing period in subjects with refractory wet age-related macular degeneration (wAMD).

Detailed Description: This study is designed to investigate the potential therapeutic effects and safety of oral ALK4290 administered 800 mg daily over a 6-week dosing period as measured by Best Corrected Visual Acuity (BCVA) in subjects with refractory wAMD (i.e., following monthly intravitreal \[IVT\] anti-VEGF therapy for at least 3 months in the study eye).

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 26

Inclusion Criteria

Men and women with refractory active CNV secondary to AMD, diagnosed by a retinal specialist with all the following characteristics and ophthalmic inclusion criteria applied to the study eye:
- Persistent exudation of SRF and IRF as documented by SD-OCT and absence of improvement in visual acuity following monthly IVT anti-VEGF therapy of at least 3 months; subject must have received their last IVT anti-VEGF injection 30 to 90 days prior to the initial screening visit
- Central subfield retinal thickness ≥ 250 microns on SD-OCT (exclusive of subretinal pigment epithelial fluid, inclusive of SRF)
- Total lesion size not greater than 12 disc areas on FA
- If present, subretinal hemorrhage must comprise < 50% of the total lesion area on FA
- No subfoveal fibrosis or atrophy on FA
BCVA letter score, as measured by ETDRS in the study eye, between 70 and 24 letters, inclusive, at screening
Patients 50 years of age or older at screening visit 1
Body mass index (BMI) between18 and ≤ 40 at screening visit 1
Female subjects must not be pregnant or breastfeeding. Women of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in the study, she should inform her treating physician immediately
Signed informed consent consistent with ICH-GCP guidelines and local legislation prior to participation in the trial, which includes medication washout and restrictions

Exclusion Criteria

Treatment with IVT anti-VEGF therapy within 30 days preceding screening visit in the study eye and/or planned concomitant IVT anti-VEGF treatment in the fellow eye during the study period
Previous participation in any studies of investigational drugs within 1 month preceding screening visit
Any form of macular degeneration that is not age-related (e.g., Best's disease, Stargardt's disease, Sorsby's disease, etc.)
Additional eye disease in the study eye that could compromise BCVA (i.e., uncontrolled glaucoma (intraocular pressure > 24) with visual field loss, clinically significant diabetic maculopathy, history of ischemic optic neuropathy or retinal vascular occlusion, vitreomacular traction, monocular vision, or genetic disorders such as retinitis pigmentosa; high myopia > 8 diopters)
Anterior segment and vitreous abnormalities in the study eye that would preclude adequate observation with fundus photography/FA or SD-OCT
Intraocular surgery in the study eye within 3 months prior to screening
Aphakia or total absence of the posterior capsule (yttrium aluminum garnet (YAG) laser capsulotomy permitted, a minimum of 1 month prior to enrollment) in the study eye

Study & Design

Study Type: INTERVENTIONAL

Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Active	ALK4290	ALK4290 800 mg daily

Primary Outcome Measures

Name	Time	Method
Best Corrected Visual Acuity (BCVA)	Baseline to 6 weeks	Mean changes in BCVA as measured by the Early Treatment Diabetic Retinopathy Study (ETDRS) testing method. The ETDRS chart assesses changes in vision based on the number of letters correctly read by the subject

Secondary Outcome Measures

Name	Time	Method
Incidence of Treatment-emergent Adverse Events (Safety)	Baseline to 10 weeks	Treatment-emergent adverse events identified by the Common Terminology Criteria for Adverse Events (CTCAE v4.03)

Trial Locations

Locations (5): Jahn Ferenc South-Pest Hospital and Clinic
🇭🇺
Budapest, Hungary
Markusovszky University Teaching Hospital
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Szombathely, Hungary
Borsod-Abauj-Zemplen County Hospital and Teaching Hospital
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Miskolc, Hungary
Szabolcs-Szatmar-Bereg County Hospital and University Hospital
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Nyíregyháza, Hungary
University of Szeged Faculty of Medicine
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Szeged, Hungary